Development and biological characterization of a clinical gene transfer vector for the treatment of MAK-associated retinitis pigmentosa
Gene Therapy, Published online: 14 September 2021; doi:10.1038/s41434-021-00291-5Development and biological characterization of a clinical gene transfer vector for the treatment of MAK-associated retinitis pigmentosa (Source: Gene Therapy)
Source: Gene Therapy - September 14, 2021 Category: Genetics & Stem Cells Authors: Budd A. Tucker Erin R. Burnight Cathryn M. Cranston Mallory J. Ulferts Meagan A. Luse Trudi Westfall C. Anthony Scott Autumn Marsden Katherine Gibson-Corley Luke A. Wiley Ian C. Han Diane C. Slusarski Robert F. Mullins Edwin M. Stone Source Type: research
Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)
Gene Therapy, Published online: 08 September 2021; doi:10.1038/s41434-021-00284-4Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI) (Source: Gene Therapy)
Source: Gene Therapy - September 8, 2021 Category: Genetics & Stem Cells Authors: Jennifer E. Adair Lindsay Androski Lois Bayigga Deus Bazira Eugene Brandon Lynda Dee Steven Deeks Mohammed Draz Karine Dub é Mark Dybul Umut Gurkan Evelyn Harlow Cissy Kityo Michael Louella Punam Malik Vikram Mathews Adrian McKemey Henry Mugerwa Daniel M Source Type: research
Ultra-sensitive AAV capsid detection by immunocapture-based qPCR following factor VIII gene transfer
Gene Therapy, Published online: 23 August 2021; doi:10.1038/s41434-021-00287-1Ultra-sensitive AAV capsid detection by immunocapture-based qPCR following factor VIII gene transfer (Source: Gene Therapy)
Source: Gene Therapy - August 23, 2021 Category: Genetics & Stem Cells Authors: Krystal Sandza Annie Clark Elli Koziol Hassibullah Akeefe Fan Yang Jennifer Holcomb Kathryn Patton Kevin Hammon Nina Mitchell Wing Y. Wong Stephen J. Zoog Benjamin Kim Joshua Henshaw Christian Vettermann Source Type: research
CCR1 enhances SUMOylation of DGCR8 by up-regulating ERK phosphorylation to promote spinal nerve ligation-induced neuropathic pain
Gene Therapy, Published online: 20 August 2021; doi:10.1038/s41434-021-00285-3CCR1 enhances SUMOylation of DGCR8 by up-regulating ERK phosphorylation to promote spinal nerve ligation-induced neuropathic pain (Source: Gene Therapy)
Source: Gene Therapy - August 20, 2021 Category: Genetics & Stem Cells Authors: Cunxian Shi Jin Jin Hongyu Xu Jiahai Ma Tao Li Yonggang Xie Zhen Li Source Type: research
Novel design of nucleic acid standards for hydrolysis probe-based PCR with melting analysis
Gene Therapy, Published online: 19 August 2021; doi:10.1038/s41434-021-00288-0Novel design of nucleic acid standards for hydrolysis probe-based PCR with melting analysis (Source: Gene Therapy)
Source: Gene Therapy - August 19, 2021 Category: Genetics & Stem Cells Authors: Anna Baoutina Somanath Bhat Source Type: research
Characterization of integration frequency and insertion sites of adenovirus DNA into mouse liver genomic DNA following intravenous injection
Gene Therapy, Published online: 18 August 2021; doi:10.1038/s41434-021-00278-2Characterization of integration frequency and insertion sites of adenovirus DNA into mouse liver genomic DNA following intravenous injection (Source: Gene Therapy)
Source: Gene Therapy - August 18, 2021 Category: Genetics & Stem Cells Authors: Zhibin Wang Philip J. Troilo Thomas G. Griffiths II Laural B. Harper Amy B. Barnum Stephen J. Pacchione Cindy J. Pauley Jose A. Lebron Jayanthi Wolf Brian J. Ledwith Source Type: research
Thrombopoietin-based CAR-T cells demonstrate in vitro and in vivo cytotoxicity to MPL positive acute myelogenous leukemia and hematopoietic stem cells
Gene Therapy, Published online: 13 August 2021; doi:10.1038/s41434-021-00283-5Thrombopoietin-based CAR-T cells demonstrate in vitro and in vivo cytotoxicity to MPL positive acute myelogenous leukemia and hematopoietic stem cells (Source: Gene Therapy)
Source: Gene Therapy - August 13, 2021 Category: Genetics & Stem Cells Authors: Jaquelyn T. Zoine Chengyu Prince Jamie Y. Story Gianna M. Branella Allison M. Lytle Andrew Fedanov Jordan S. Alexander Christopher C. Porter Christopher B. Doering H. Trent Spencer Shanmuganathan Chandrakasan Source Type: research
Non-viral delivery of CRISPR–Cas9 complexes for targeted gene editing via a polymer delivery system
Gene Therapy, Published online: 06 August 2021; doi:10.1038/s41434-021-00282-6Non-viral delivery of CRISPR–Cas9 complexes for targeted gene editing via a polymer delivery system (Source: Gene Therapy)
Source: Gene Therapy - August 6, 2021 Category: Genetics & Stem Cells Authors: Jonathan O ’Keeffe Ahern Irene Lara-S áez Dezhong Zhou Rodolfo Murillas Jose Bonafont Ángeles Mencía Marta Garc ía Dar ío Manzanares Jennifer Lynch Ruth Foley Qian Xu A Sigen Fernando Larcher Wenxin Wang Source Type: research
XIAP gene therapy effects on retinal ganglion cell structure and function in a mouse model of glaucoma
Gene Therapy, Published online: 06 August 2021; doi:10.1038/s41434-021-00281-7XIAP gene therapy effects on retinal ganglion cell structure and function in a mouse model of glaucoma (Source: Gene Therapy)
Source: Gene Therapy - August 6, 2021 Category: Genetics & Stem Cells Authors: Shagana Visuvanathan Adam N. Baker Pamela S. Lagali Stuart G. Coupland Garfield Miller William W. Hauswirth Catherine Tsilfidis Source Type: research
Suppression of bone metastatic castration-resistant prostate cancer cell growth by a suicide gene delivered by JC polyomavirus-like particles
Gene Therapy, Published online: 21 July 2021; doi:10.1038/s41434-021-00280-8Suppression of bone metastatic castration-resistant prostate cancer cell growth by a suicide gene delivered by JC polyomavirus-like particles (Source: Gene Therapy)
Source: Gene Therapy - July 21, 2021 Category: Genetics & Stem Cells Authors: Cheng-Huang Shen Mien-Chun Lin Chiung-Yao Fang Pei-Lain Chen Chih-Chieh Chou Chun-Liang Tung Chun-Nun Chao Deching Chang Meilin Wang Source Type: research
Screening for gene doping transgenes in horses via the use of massively parallel sequencing
Gene Therapy, Published online: 19 July 2021; doi:10.1038/s41434-021-00279-1Screening for gene doping transgenes in horses via the use of massively parallel sequencing (Source: Gene Therapy)
Source: Gene Therapy - July 19, 2021 Category: Genetics & Stem Cells Authors: Jillian Maniego Bogusia Pesko Jocelyn Habershon-Butcher Jim Huggett Polly Taylor James Scarth Edward Ryder Source Type: research
Pulmonary transplantation of alpha-1 antitrypsin (AAT)-transgenic macrophages provides a source of functional human AAT in vivo
Gene Therapy, Published online: 19 July 2021; doi:10.1038/s41434-021-00269-3Pulmonary transplantation of alpha-1 antitrypsin (AAT)-transgenic macrophages provides a source of functional human AAT in vivo (Source: Gene Therapy)
Source: Gene Therapy - July 19, 2021 Category: Genetics & Stem Cells Authors: Ewa Janosz Miriam Hetzel Hanna Spielmann Srinu Tumpara Charlotte Rossdam Marc Schwabbauer Doreen Kloos Constantin von Kaisenberg Axel Schambach Falk F. R. Buettner Sabina Janciauskiene Nico Lachmann Thomas Moritz Source Type: research
Gene therapy for spinal muscular atrophy: the Qatari experience
Gene Therapy, Published online: 19 July 2021; doi:10.1038/s41434-021-00273-7Gene therapy for spinal muscular atrophy: the Qatari experience (Source: Gene Therapy)
Source: Gene Therapy - July 19, 2021 Category: Genetics & Stem Cells Authors: Hossamaldein Gaber Ali Khalid Ibrahim Mahmoud Fawzi Elsaid Reem Babiker Mohamed Mahmoud I. A. Abeidah Azhar Othman Al Rawwas Khaled Elshafey Hajer Almulla Karen El-Akouri Mariam Almulla Amna Othman Sara Musa Fatma Al-Mesaifri Rehab Ali Noora Shahbeck Mari Source Type: research
Restoration of RPGR expression in vivo using CRISPR/Cas9 gene editing
Gene Therapy, Published online: 14 July 2021; doi:10.1038/s41434-021-00258-6Restoration of RPGR expression in vivo using CRISPR/Cas9 gene editing (Source: Gene Therapy)
Source: Gene Therapy - July 14, 2021 Category: Genetics & Stem Cells Authors: Jessica D. Gumerson Amal Alsufyani Wenhan Yu Jingqi Lei Xun Sun Lijin Dong Zhijian Wu Tiansen Li Source Type: research
Viral vector gene delivery of the novel chaperone protein SRCP1 to modify insoluble protein in in vitro and in vivo models of ALS
Gene Therapy, Published online: 08 July 2021; doi:10.1038/s41434-021-00276-4Viral vector gene delivery of the novel chaperone protein SRCP1 to modify insoluble protein in in vitro and in vivo models of ALS (Source: Gene Therapy)
Source: Gene Therapy - July 8, 2021 Category: Genetics & Stem Cells Authors: Ian W. Luecke Gloria Lin Stephanie Santarriaga K. Matthew Scaglione Allison D. Ebert Source Type: research
